AR119430A1 - Métodos para tratar o prevenir la atrofia muscular espinal - Google Patents
Métodos para tratar o prevenir la atrofia muscular espinalInfo
- Publication number
- AR119430A1 AR119430A1 ARP200102019A ARP200102019A AR119430A1 AR 119430 A1 AR119430 A1 AR 119430A1 AR P200102019 A ARP200102019 A AR P200102019A AR P200102019 A ARP200102019 A AR P200102019A AR 119430 A1 AR119430 A1 AR 119430A1
- Authority
- AR
- Argentina
- Prior art keywords
- antisense compound
- muscular atrophy
- spinal muscular
- treat
- methods
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
- G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Dermatology (AREA)
- Food Science & Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Analytical Chemistry (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962876360P | 2019-07-19 | 2019-07-19 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR119430A1 true AR119430A1 (es) | 2021-12-15 |
Family
ID=71995097
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP200102019A AR119430A1 (es) | 2019-07-19 | 2020-07-17 | Métodos para tratar o prevenir la atrofia muscular espinal |
Country Status (31)
Country | Link |
---|---|
US (2) | US20220325282A1 (fr) |
EP (2) | EP3999643B1 (fr) |
JP (2) | JP7279253B2 (fr) |
KR (1) | KR20220042113A (fr) |
CN (1) | CN114302727A (fr) |
AR (1) | AR119430A1 (fr) |
AU (1) | AU2020316997A1 (fr) |
BR (1) | BR112022000870A2 (fr) |
CA (1) | CA3149906A1 (fr) |
CL (1) | CL2021003602A1 (fr) |
CO (1) | CO2022001354A2 (fr) |
CR (1) | CR20220071A (fr) |
DK (1) | DK3999643T5 (fr) |
ES (1) | ES2966791T3 (fr) |
FI (1) | FI3999643T3 (fr) |
HR (1) | HRP20231515T1 (fr) |
HU (1) | HUE064071T2 (fr) |
IL (1) | IL289745A (fr) |
JO (1) | JOP20220007A1 (fr) |
LT (1) | LT3999643T (fr) |
MA (1) | MA62935B1 (fr) |
MD (1) | MD3999643T2 (fr) |
MX (1) | MX2022000441A (fr) |
PE (1) | PE20220941A1 (fr) |
PL (1) | PL3999643T3 (fr) |
PT (1) | PT3999643T (fr) |
RS (1) | RS64879B1 (fr) |
SI (1) | SI3999643T1 (fr) |
TW (1) | TWI841762B (fr) |
UY (1) | UY38790A (fr) |
WO (1) | WO2021016032A1 (fr) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3797780B1 (fr) | 2014-04-17 | 2022-09-14 | Biogen MA Inc. | Compositions et procédés de modulation de l'épissage du smn2 chez un sujet |
BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
FI3999643T3 (fi) | 2019-07-19 | 2023-11-30 | Biogen Ma Inc | Menetelmiä spinaalisen lihasatrofian hoitamiseksi tai ehkäisemiseksi |
WO2024138018A1 (fr) * | 2022-12-23 | 2024-06-27 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Traitement de troubles induits par gemin5 |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
HUE027486T2 (en) | 2005-06-23 | 2016-09-28 | Isis Pharmaceuticals Inc | Preparations and methods for modifying SMN2-Splicing |
WO2008157753A1 (fr) * | 2007-06-21 | 2008-12-24 | Sma Foundation | Procédés de traitement d'une atrophie musculaire spinale |
CA2765396C (fr) | 2009-06-17 | 2019-08-20 | Isis Pharmaceuticals, Inc. | Compositions et methodes pour moduler l'epissage de smn2 chez un sujet |
WO2011032109A1 (fr) | 2009-09-11 | 2011-03-17 | Sma Foundation | Biomarqueurs de l'atrophie musculaire spinale |
EP2714037B1 (fr) * | 2011-05-25 | 2016-07-13 | Université Paris Descartes | Inhibiteurs de la voie erk pour le traitement de l'amyotrophie spinale |
EP2943225A4 (fr) * | 2013-01-09 | 2016-07-13 | Ionis Pharmaceuticals Inc | Compositions et procédés pour la modulation de l'épissage de smn2 chez un sujet |
BR112017004056A2 (pt) * | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
MX2019000619A (es) * | 2016-07-15 | 2019-08-01 | Ionis Pharmaceuticals Inc | Compuestos y metodos para la modulacion de smn2. |
TW201927241A (zh) | 2017-12-21 | 2019-07-16 | 瑞士商赫孚孟拉羅股份公司 | 用於肌肉失能之數位生物標記 |
MA51678A (fr) | 2018-01-25 | 2020-12-02 | Biogen Ma Inc | Méthodes de traitement de l'amyotrophie musculaire |
IT201800004359A1 (it) | 2018-04-10 | 2019-10-10 | IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE | |
US20210308281A1 (en) * | 2018-08-15 | 2021-10-07 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
FI3999643T3 (fi) | 2019-07-19 | 2023-11-30 | Biogen Ma Inc | Menetelmiä spinaalisen lihasatrofian hoitamiseksi tai ehkäisemiseksi |
-
2020
- 2020-07-16 FI FIEP20753556.8T patent/FI3999643T3/fi active
- 2020-07-16 EP EP20753556.8A patent/EP3999643B1/fr active Active
- 2020-07-16 JO JOP/2022/0007A patent/JOP20220007A1/ar unknown
- 2020-07-16 MX MX2022000441A patent/MX2022000441A/es unknown
- 2020-07-16 PT PT207535568T patent/PT3999643T/pt unknown
- 2020-07-16 SI SI202030323T patent/SI3999643T1/sl unknown
- 2020-07-16 MA MA62935A patent/MA62935B1/fr unknown
- 2020-07-16 CN CN202080049461.1A patent/CN114302727A/zh active Pending
- 2020-07-16 BR BR112022000870A patent/BR112022000870A2/pt unknown
- 2020-07-16 RS RS20231128A patent/RS64879B1/sr unknown
- 2020-07-16 KR KR1020227001151A patent/KR20220042113A/ko unknown
- 2020-07-16 LT LTEPPCT/US2020/042312T patent/LT3999643T/lt unknown
- 2020-07-16 DK DK20753556.8T patent/DK3999643T5/da active
- 2020-07-16 MD MDE20220574T patent/MD3999643T2/ro unknown
- 2020-07-16 AU AU2020316997A patent/AU2020316997A1/en active Pending
- 2020-07-16 CA CA3149906A patent/CA3149906A1/fr active Pending
- 2020-07-16 ES ES20753556T patent/ES2966791T3/es active Active
- 2020-07-16 WO PCT/US2020/042312 patent/WO2021016032A1/fr active Application Filing
- 2020-07-16 PL PL20753556.8T patent/PL3999643T3/pl unknown
- 2020-07-16 EP EP23210526.2A patent/EP4335503A3/fr active Pending
- 2020-07-16 CR CR20220071A patent/CR20220071A/es unknown
- 2020-07-16 PE PE2021002322A patent/PE20220941A1/es unknown
- 2020-07-16 US US17/626,324 patent/US20220325282A1/en active Pending
- 2020-07-16 HU HUE20753556A patent/HUE064071T2/hu unknown
- 2020-07-16 JP JP2022500671A patent/JP7279253B2/ja active Active
- 2020-07-16 UY UY0001038790A patent/UY38790A/es unknown
- 2020-07-16 HR HRP20231515TT patent/HRP20231515T1/hr unknown
- 2020-07-17 TW TW109124224A patent/TWI841762B/zh active
- 2020-07-17 AR ARP200102019A patent/AR119430A1/es unknown
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2021
- 2021-12-31 CL CL2021003602A patent/CL2021003602A1/es unknown
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2022
- 2022-01-10 IL IL289745A patent/IL289745A/en unknown
- 2022-02-10 CO CONC2022/0001354A patent/CO2022001354A2/es unknown
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2023
- 2023-05-10 JP JP2023077805A patent/JP2023100911A/ja active Pending
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2024
- 2024-05-10 US US18/661,339 patent/US20240318182A1/en active Pending
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